PB1125 - Musculoskeletal health and health-related quality of life in patients switching to emicizumab prophylaxis

Tuesday, July 12, 2022

6:30 PM – 7:30 PM

Location: Exhibition

Presentation Format: In-Person

Presenting Author(s)

SA

Sara Arcudi, MD

Academic researcher, MD, PhD
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi
Milan, Lombardia, Italy

Co-Author(s)

Roberta Gualtierotti, MD, PhD

Assistant Professor
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico, Centro Emofilia e Trombosi Angelo Bianchi Bonomi
Milan, Lombardia, Italy
VB

Valentina Begnozzi

Physioterapist
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milano, Lombardia, Italy
AC

Alessandro Ciavarella

PhD Student
Università degli Studi di Milano, Department of Biomedical Sciences for Health
Milan, Lombardia, Italy
GN

Gabriella Nicolò

Research Nurse
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano, Department of Healthcare Professions, Milan, Italy
Milano, Lombardia, Italy
SS

Simona Maria Siboni

Hematologist
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center
Milan, Lombardia, Italy
Eugenia Biguzzi, MD

Hematologist
Hematology clinic, Oncology Institute of Southern Switzerland, EOC
Bellinzona, Ticino, Switzerland
EB

Elena Boccalandro

Physiotherapist
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Milan, Italy
Milan, Lombardia, Italy
Flora Peyvandi, MD, PhD

Professor of Internal Medicine; Director of the Angelo Bianchi Bonomi Hemophilia & Thrombosis Center
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy
Milan, Lombardia, Italy

Background: Emicizumab is effective as a non-replacement prophylaxis for patients with severe hemophilia A, but its effect on musculoskeletal health is still under debate.

Aims: To explore the effect of emicizumab prophylaxis on musculoskeletal health and health-related quality of life (HRQoL) in patients affected with severe hemophilia A (HA) from the Milan cohort.

Methods: We collected hemophilia joint health score (HJHS), the five-level-EQ-5D (EQ-5D-5L) and hemophilia early arthropathy detection with ultrasound (HEAD-US) in patients with HA switched to emicizumab in the post-marketing era, at baseline before switching and after a follow-up of at least 8 months. The HJHS was performed by a trained physiotherapist and the HEAD-US was performed on the same joints by an expert rheumatologist. Paired sample T-test was used to assess differences before and after switching.

Results: We evaluated 8 consecutive adults at baseline and 8-12 months after switching to emicizumab prophylaxis. Mean age was 43.3 years (SD 17.1). Three out of 8 patients had one or more prosthetic joints. Mean±SD HEAD-US total score was stable (13±9.6 to 12.5±8.7, p-value 0.5), with a slight improvement of 1 to 2 points in total synovitis domain in 3 out of 8 patients. Mean±SD HJHS decreased from 9.6±9.2 to 7.6±8.1 (p-value < 0.05). When analyzing the pain/discomfort domain of the EQ-5D-5L, an improvement was reported in 5 out of 8 patients from “slight problems” to “no problems” after switching, two patients remained at the same level and one patient improved from “severe” to “moderate problems”.

Conclusion(s): Our preliminary results showed improved or stable musculoskeletal health in patients switched to emicizumab. As expected, the cartilage and bone damage domains of the HEAD-US score do not seem to be influenced by the therapy switch, whereas an improvement in synovitis, HJHS score and HRQoL was observed.